ong-Term Evaluation of BIIB067
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)MedDRA version: 20.0Level: PTClassification code 10077024Term: Familial amyotrophic lateral sclerosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2016-003225-41-BE
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 183
- Must have diagnosis of SOD1-ALS, and must have completed the End of Study Visit for either Part A, B and/or Part C of Study 233AS101 (i.e., were not withdrawn).
- If taking riluzole, must be receiving a stable dose for =30 days prior to Day 1.
- For female participants of childbearing potential must agree to practice effective contraception during the study and for 5 months after their last dose of study treatment.
- Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
- Participants from Study 233AS101 Parts A and B must have a washout =16 weeks between the last dose of study treatment received in Study 233AS101 and the first dose of BIIB067 received in the current Study 233AS102.
- If taking edaravone, participant must have initiated edaravone = 60 days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing days.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 146
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 37
Key Exclusion Criteria:
- History of allergies to a broad range of anaesthetics
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after an LP procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g. vascular abnormalities, neoplams, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g. hemophilia, Von Willebrand's disease, liver disease).
- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.
- Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
- Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.
- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
- Female subjects who are pregnant or currently breastfeeding.
- Current enrollment in any other interventional study.
- Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis (N4-methylthiosemicarbazone)) or pyrimethamine.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation.;Secondary Objective: The secondary objective is to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) profiles and effects on disease progression of BIIB067 administered to subjects with ALS and confirmed SOD1 mutation.;Primary end point(s): - Number of participants experiencing AEs and serious adverse events (SAEs)<br>;Timepoint(s) of evaluation of this end point: Up to Week 364
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Levels of BIIB067 in Plasma<br>- Levels of BIIB067 in Cerebral Spinal Fluid (CSF)<br>- Change from baseline in Total SOD1 Protein in CSF<br>- Change from baseline in Neurofilament Light Chain (NfL) Concentration in Plasma<br>- Change from Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Score<br>- Change in Basline in Slow Vitcal Capacity (SVC)<br>- Change in Baseline in Handheld Dynamotry (HHD) MegaScore and Individual Muscle Strength<br>- Time to death or Permanent Ventilation<br>- Time to death;Timepoint(s) of evaluation of this end point: Up to week 364